Latest news with #SMA
The Hindu
6 hours ago
- Entertainment
- The Hindu
Shankar Mahadevan Academy turns 15
At a press meet at the South Asia Institute of Advanced Christian Studies Centre (SAIACS) in Bengaluru, the air buzzed with more than just media chatter. It carried echoes of swaras, stories, and something deeply personal. 'We never knew this day would come so fast,' said Shankar Mahadevan, eyes crinkling with joy, reflecting on the 15-year journey of the Shankar Mahadevan Academy (SMA) — a dream that started with 15 students, most of them his nieces and nephews, and is today a global musical movement spanning 94 countries. 'It felt like a reunion of purpose,' said the Palakkad-based singer and composer. The Shankar Mahadevan Academy, founded in 2011 by Shankar and technology entrepreneur Sridhar Ranganathan, was a pioneer of sorts, attempting to do something unique at the time: offering online music education. 'People laughed. Music? Online? Will it even work?' Shankar recalled. It did not just work, it soared. Today, over 50,000 students have learned through the academy's unique digital platform, with more than half a million live classes taught. Numbers tell only part of the story. To celebrate its 15th year, the academy is launching courses that go far beyond traditional syllabi, including Garbh Sangeet, a course for expecting mothers that uses classical ragas to create emotional and spiritual connections before birth. 'Inside the academy, we call it 'minus one to infinity, '' smiled Ranganathan. The poetic phrase captures a powerful belief — that music is not just for learning or performing, but for living, healing, and bonding. Another offering, the Playback Singing Series, is equally ambitious — a rigorous, multi-year training program that fuses classical fundamentals with film music, preparing aspirants for the nuanced world of playback singing. Over the years, SMA has expanded into other avenues, including Sangam, a student-teacher music festival, and Prayag, an elite stage for dedicated learners. 'These are not just students. They are sadhakas,' said Shankar. 'This is not just education — it's an emotional legacy.' From children barely old enough to walk, to 70-year-olds who have never sung before, SMA has bridged generations. 'When I see a grandfather in Toronto learning alongside his granddaughter in Bengaluru… It's magical,' Mahadevan shared. The academy has also given back, launching initiatives including SMA Nirvana — live musical performances streamed to patients in hospitals and hospices, SMA Muskara, a pension program for aged or injured musicians, and Joyful Choir, an inclusive initiative for children on the autism spectrum. Their nonprofit wing has helped bring music education to children in Dharavi, Goa, and now, through their newest initiative — Reach Out India — to students in remote villages via internet-powered classrooms. 'One teacher in Ahmedabad is teaching kids in Kumbakonam — and they all performed for me,' said Shankar , admitting that the journey has given him a deeper sense of fulfilment. 'I've always known music entertains. But through this academy, I learnt that music can transform.'
Yahoo
a day ago
- Health
- Yahoo
Biogen's Spinraza successor advances to Phase III
Biogen's antisense oligonucleotide (ASO), salanersen, is advancing to Phase III trials after it showed benefit with a lower dosing schedule than Spinraza (nusinersen) in patients with spinal muscular atrophy (SMA). Leveraging the same mechanism of action as Spinraza but designed to achieve greater potency, Biogen believes that salanersen has the potential to achieve high efficacy while enabling once yearly dosing. Meanwhile, Spinraza is dosed once every four months. Interim analysis of the Phase I study (NCT05575011) found that both dose levels tested, 40mg and 80mg, given once a year, were generally well-tolerated and led to substantial slowing of neurodegeneration. This was shown by a 70% mean reduction in neurofilament light chain (NfL) at six months. Half of the patients dosed with salanersen achieved new WHO motor milestones that they previously could not achieve on their own or required assistance to do, such as walking, crawling, standing, or sitting. These patients also experienced clinically meaningful improvements in motor function from baseline to one year, including a 3.3-point mean improvement from baseline on the Hammersmith Functional Motor Scale – Expanded (HFMSE) and a 5.3-point improvement on the Revised Upper Limb Module (RULM). Salanersen was generally well tolerated in both doses, with most adverse events (AEs) mild to moderate in severity. The most common AEs were pyrexia and upper respiratory tract infection. The interim analysis comes from Part B of the study, an open-label segment that enrolled paediatric SMA patients who previously received Novartis' Zolgensma (onasemnogene abeparvovec) and had investigator-reported suboptimal clinical status. The data is set to be presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Anaheim, California, on 25 June. Biogen is now engaging with global health authorities to initiate Phase III studies. Biogen licensed the global development, manufacturing and commercialisation rights for salanersen from Ionis Pharmaceuticals. This is the second SMA collaboration between the pair, with Spinraza also originally coming from Ionis. GlobalData analysis shows that Spinraza made $1.57bn in sales in 2024, with 2030 sales projected to reach $1.19bn. This drop in sales is due in part to the therapy losing its US exclusivity in December 2023. GlobalData is the parent company of Clinical Trials Arena. Principal investigator for the salanersen Phase I trial and Clinical Center NeMO's clinical and scientific director Dr Valeria A Sansone said: 'Of the data generated, to me, it is neurofilament and the WHO milestones that are most easily interpretable, given these children had previously received gene therapy. To see a child dosed with gene therapy at one year of age and still unable to sit without support at age five, then gain the ability to sit independently just three months after initiating salanersen, that is unexpected.' SMA is a rare, genetic and neuromuscular disease that affects patients of all ages. It is characterised by a loss of motor neurons in the spinal cord and lower brain stem, resulting in progressive muscle atrophy and weakness. Roche's Evrysdi (risdiplam) is also approved for use in SMA, having gained US Food and Drug Administration (FDA) approval in August 2020. Scholar Rock's apetigromab has received priority review from the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of 22 September 2025. According to GlobalData analysis, the SMA market across the seven major markets (7MM: France, Germany, Italy, Japan, Spain, the UK and the US) is set to grow from $2.7bn in 2023 to $3bn in 2033. "Biogen's Spinraza successor advances to Phase III" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
Yahoo
a day ago
- Business
- Yahoo
Ionis announces Biogen to advance salanersen into SMA registrational studies based on positive interim Phase 1 results
– Investigational salanersen (ION306/BIIB115) developed using novel Ionis antisense chemistry with the potential to achieve high efficacy and once-yearly dosing – – Interim Phase 1 data show children with SMA previously treated with gene therapy experienced a substantial slowing of neurodegeneration and clinically meaningful improvements in motor function following initiation of salanersen – – Biogen is engaging with regulators to advance salanersen to registrational stage studies – CARLSBAD, Calif., June 25, 2025--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that its partner, Biogen, shared positive topline results from the Phase 1 study of salanersen (ION306/BIIB115), an investigational antisense oligonucleotide (ASO) being developed for the potential treatment of spinal muscular atrophy (SMA). Leveraging the same mechanism of action as SPINRAZA® (nusinersen) but designed to achieve greater potency, salanersen has the potential to achieve high efficacy and enable once-yearly dosing. Both dose levels tested, 40 mg and 80 mg, given once a year, were generally well-tolerated and led to substantial slowing of neurodegeneration, as shown by reductions in neurofilament. Exploratory clinical outcome data show clinically meaningful improvements in function and attainment of new World Health Organization (WHO) milestones over one year. These data will be presented today at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Anaheim, Calif. The Phase 1 single ascending dose study was designed to evaluate the safety, tolerability and pharmacokinetics of salanersen. The trial consisted of two parts: Part A, a randomized and placebo-controlled segment in healthy adult male volunteers and Part B, an open-label segment in pediatric participants with SMA who previously received ZOLGENSMA® (onasemnogene abeparvovec) and had investigator-reported suboptimal clinical status. Interim results are from Part B (n=24) in individuals who received either 40 mg or 80 mg salanersen once a year. In participants with elevated baseline concentrations of neurofilament light chain (NfL), indicating ongoing neurodegeneration, initiation of salanersen led to mean reductions in NfL of 70% at 6-months which were sustained through the one-year dosing interval. "These encouraging interim results reflect the potential of Ionis' leading technology to continue to improve the lives of people living with SMA," said Holly Kordasiewicz, Ph.D., senior vice president of neurology, Ionis. "We're proud to have discovered salanersen, which builds on the foundation we established with SPINRAZA and further supports Ionis' long-standing leadership in neurology and innovative RNA-targeted medicines. We are deeply grateful to the patients, families and investigators who participated in this study and look forward to continuing to work with Biogen to support the SMA community." Salanersen was invented by Ionis using a novel antisense chemistry designed to enhance potency, stability and durability. This advanced molecular design has the potential to deliver long-term results with long-interval dosing. In addition to safety and NfL, exploratory clinical outcome data were evaluated for the subgroup of participants with at least one year of follow-up at the time of the interim analysis (n=8 participants aged 2-12 who received 40 mg of salanersen). Half (4/8) of these participants achieved new WHO motor milestones that they previously could not achieve on their own or for which they required assistance, such as walking, crawling, standing or sitting. Furthermore, these participants experienced clinically meaningful improvements in motor function from baseline to one year, including a 3.3-point (SD 4.46) mean improvement from baseline on the Hammersmith Functional Motor Scale – Expanded (HFMSE) and a 5.3-point (SD 4.75) improvement on the Revised Upper Limb Module (RULM). "Despite the remarkable therapeutic advancements in the field of SMA over the past decade, there remains critical unmet needs. Salanersen represents the next phase of Biogen's ongoing pursuit to address these needs," said Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen. "We are encouraged by the available data and eager to move salanersen into the next stage of development as quickly as possible. We are deeply grateful for the trial participants and their families, investigators, and site staff." The cumulative interim data from the Phase 1 study indicate that salanersen was generally well tolerated at the 40 mg and 80 mg doses, with most adverse events (AEs) mild to moderate in severity. The most common AEs were pyrexia and upper respiratory tract infection. Biogen is currently engaging with global health authorities regarding the design of the Phase 3 studies. Ionis discovered salanersen and licensed the global development, manufacturing and commercialization rights to Biogen Inc. About Spinal Muscular Atrophy (SMA) SMA is a rare, genetic, neuromuscular disease that affects individuals of all ages. It is characterized by a loss of motor neurons in the spinal cord and lower brain stem, resulting in progressive muscle atrophy and weakness. SMA is caused by a deficiency in the production of survival motor neuron (SMN) protein due to a damaged or missing SMN1 gene, with a spectrum of disease severity. Some individuals with SMA may never sit; some sit but never walk; and some walk but may lose that ability over time. In the absence of treatment, children with the most severe form of SMA would usually not be expected to reach their second birthday. SMA impacts approximately 1 in 10,000 live births, is a leading cause of genetic death among infants and causes a range of disability in teenagers and adults. About SPINRAZA SPINRAZA® (nusinersen) 12 mg/5 mL injection is approved in more than 71 countries to treat infants, children and adults with spinal muscular atrophy (SMA). As a foundation of care in SMA, more than 14,000 individuals have been treated with SPINRAZA worldwide. SPINRAZA is an antisense oligonucleotide (ASO) that targets the underlying cause of motor neuron loss by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body. It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts. SPINRAZA has shown efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 10 years, combined with unsurpassed real-world experience. The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs. Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country's product website. About Ionis Neurology Ionis has been at the forefront of discovering and developing leading neurological disease medicines, including SPINRAZA® (nusinersen), the first approved treatment for spinal muscular atrophy, WAINUA™ (eplontersen), a medicine to treat hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), and QALSODY® (tofersen) for SOD1-ALS. The clinical-stage portfolio includes 13 therapies, of which eight are wholly owned by Ionis. Ionis' investigational portfolio includes medicines for which there are few or no disease modifying treatments, such as rare diseases including Angelman syndrome, Prion disease and Alexander disease and more common conditions such as Alzheimer's and Parkinson's disease. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has six marketed medicines and a leading pipeline in neurology, cardiology, and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit and follow us on X (Twitter), LinkedIn and Instagram. Ionis Forward-looking Statements This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of salanersen (ION306/BIIB115), our commercial medicines, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc. SPINRAZA® and QALSODY® are registered trademarks of Biogen. WAINUA™ is a registered trademark of the AstraZeneca group of companies. View source version on Contacts Ionis Investor Contact: D. Wade Walke, 760-603-2331 Ionis Media Contact: Hayley Soffermedia@ 760-603-4679 Sign in to access your portfolio
Associated Press
a day ago
- Health
- Associated Press
New Data for Nusinersen Underscore Biogen's Commitment to Advancing Clinical Research to Improve Outcomes in SMA
CAMBRIDGE, Mass., June 27, 2025 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced new data that reinforce the clinical impact of nusinersen across a broad spectrum of individuals affected by spinal muscular atrophy (SMA). These latest findings from Part C of the DEVOTE trial evaluating a higher dose regimen of nusinersen and the NURTURE trial which evaluated the approved 12 mg regimen (SPINRAZA®) in clinically presymptomatic SMA were presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Anaheim, Calif. Biogen's applications for the higher dose regimen of nusinersen are currently under review in the U.S., Europe, Japan and other global markets. The higher dose regimen of nusinersen comprises a more rapid loading regimen – two 50 mg doses 14 days apart – and a higher maintenance regimen – 28 mg every four months. 'As the SMA treatment landscape continues to evolve, we remain steadfast in our commitment to address the unmet needs of the community. The findings from Part C of the DEVOTE study further strengthen the growing body of evidence supporting the potential benefits of the higher dose regimen of nusinersen,' said Stephanie Fradette, Pharm.D., Head of the Neuromuscular Development Unit at Biogen. Improvements Observed With Higher Dose Regimen of Nusinersen in Previously Treated Patient Population Detailed results from Part C of the DEVOTE study highlight the potential clinical benefits of an investigational higher dose of nusinersen in a broad range of individuals (n=38) who had been previously treated with nusinersen at the approved 12 mg dose for approximately four years (median: 3.9 years). Participants were 4 to 65 years of age and half (n=19) were ambulatory. Participants in Part C received one loading dose (50 mg) and two maintenance doses (28 mg each) of open-label higher dose nusinersen during the study period. Most participants experienced improvements on the Hammersmith Functional Motor Scale – Expanded (HFMSE), Revised Upper Limb Module (RULM), and/or Clinical Global Impression of Change (CGI-C; assessed by investigator or caregiver) after transitioning to the higher dose regimen. These improvements were observed across phenotypes, functional status and age. For example, non-ambulatory participants improved by +2.5 (95% CI: 0.49, 4.56) on average on the HFMSE, and ambulatory participants improved by +1.1 (95% CI: -0.68, 2.89). 'These emerging data indicate that additional gains in function might be possible even in those with established disease who have been on therapy for years,' said Richard Finkel, M.D., director, Center for Experimental Neurotherapeutics (CENT) at St. Jude Children's Research Hospital. 'This effort to optimize the dosing of SPINRAZA is very exciting for the field and could fundamentally change how we treat our patients.' The safety profile of the higher dose regimen of nusinersen is broadly consistent with the known safety profile of 12 mg SPINRAZA. In the DEVOTE study overall, reported adverse events (AEs) included pneumonia, respiratory failure, pyrexia, COVID, upper respiratory tract infection, procedural pain and procedural headache. In Part C (n=40), AEs were reported in 37/40 participants, the majority of which were mild or moderate in severity. Serious AEs were reported by six participants (15%), none of which were considered by the investigator to be related to study treatment or administration. Final NURTURE Data Redefine Expectations for Early Treatment Final data from the eight-year, open-label NURTURE study highlight the impact of early intervention with 12 mg SPINRAZA in clinically presymptomatic infants with SMA. At the study conclusion, all children who participated in NURTURE (n=25) were alive and experienced continued clinical benefits over the course of the study. No participants required permanent ventilation, and the majority (20 of 25 participants) went without any ventilatory support throughout the study. Ninety-two percent of participants achieved the ability to walk independently, many within normal developmental timeframes. Participants with elevated levels of neurofilament light chain (NfL) at baseline experienced rapid and sustained reductions in NfL after initiation of nusinersen, reinforcing the potential utility of NfL as an objective biomarker of disease activity and treatment response in SMA. Nusinersen was generally well tolerated with no new safety concerns identified with eight years of follow-up. All participants had at least one AE, the majority of which were mild to moderate in severity; no AEs led to treatment discontinuation or study withdrawal. About SPINRAZA SPINRAZA (nusinersen) 12 mg/5 mL injection is approved in more than 71 countries to treat infants, children and adults with spinal muscular atrophy (SMA). As a foundation of care in SMA, more than 14,000 individuals have been treated with SPINRAZA worldwide.1 The currently approved 12 mg regimen for SPINRAZA is comprised of four loading doses administered over approximately 60 days, followed by maintenance dosing every four months thereafter. SPINRAZA is an antisense oligonucleotide (ASO) that targets the underlying cause of motor neuron loss by continuously increasing the amount of full-length survival motor neuron (SMN) protein produced in the body.2 It is administered directly into the central nervous system, where motor neurons reside, to deliver treatment where the disease starts.2 SPINRAZA has shown sustained efficacy across ages and SMA types with a well-established safety profile based on data in patients treated up to 10 years,3,4 combined with unsurpassed real-world experience. The nusinersen clinical development program encompasses more than 10 clinical studies, which have included more than 460 individuals across a broad spectrum of patient populations, including two randomized controlled studies (ENDEAR and CHERISH). The most common adverse events observed in clinical studies were respiratory infection, fever, constipation, headache, vomiting and back pain. Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs. Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq: IONS). Please click here for Important Safety Information and full Prescribing Information for SPINRAZA in the U.S., or visit your respective country's product website. About Biogen Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patients' lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth. We routinely post information that may be important to investors on our website at Follow us on social media - Facebook, LinkedIn, X, YouTube. Biogen Safe Harbor This news release contains forward-looking statements, including related to the potential clinical effects of a higher dose regimen of nusinersen; the potential benefits, safety and efficacy of higher dose regimen of nusinersen; the clinical development program for higher dose regimen of nusinersen; the identification and treatment of SMA; our research and development program for the treatment of SMA; the potential of our commercial business and pipeline programs, including SPINRAZA; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by words such as 'aim,' 'anticipate,' 'believe,' 'could,' 'estimate,' 'expect,' 'forecast,' 'intend,' 'may,' 'plan,' 'potential,' 'possible,' 'will,' 'would' and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on our forward-looking statements. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation, uncertainty of success in the development and potential commercialization of higher dose regimen of nusinersen; the risk that we may not fully enroll our clinical trials or enrollment will take longer than expected; unexpected concerns may arise from additional data, analysis or results obtained during our clinical trials; regulatory authorities may require additional information or further studies, or may fail or refuse to approve or may delay approval of our drug candidates, including SPINRAZA; the occurrence of adverse safety events; the risks of unexpected hurdles, costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; product liability claims; results of operations and financial condition. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements. References:
Hans India
a day ago
- Entertainment
- Hans India
Shankar Mahadevan Academy celebrates 15 years of spreading ‘joy of music'
Bengaluru: Shankar Mahadevan Academy (SMA), an online music education, proudly marks 15 years of bringing the joy of Indian music to learners across the globe. What began in 2011 as a bold experiment by legendary musician Shankar Mahadevan and technology leader Sridhar Ranganathan has grown into a global movement — touching lives in over 95 countries, with more than 50,000 students and over half a million live online classes conducted. Born out of passion and a belief that music knows no boundaries, SMA was founded at a time when virtual learning was still in its infancy and internet infrastructure posed significant limitations. Despite the odds, the vision was clear: to make high-quality Indian music education accessible to anyone, anywhere. Shankar Mahadevan, Co-Founder and Artistic Director of the SMA said, 'Our vision was always clear. We wanted Indian music in every home — not just in India, but around the world. Today, when I see a 65-year-old grandfather in Toronto learning alongside his 7-year-old granddaughter in Bengaluru, both discovering the same raga, it feels magical. It feels meant to be.'
